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Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-In-Human Trial.
Davies, Jane C; Polineni, Deepika; Boyd, A Christopher; Donaldson, Scott; Gill, Deborah R; Griesenbach, Uta; Hyde, Stephen C; Jain, Raksha; McLachlan, Gerry; Mall, Marcus A; Alton, Eric Wfw.
Afiliación
  • Davies JC; Imperial College London National Heart and Lung Institute, London, United Kingdom of Great Britain and Northern Ireland.
  • Polineni D; UK Respiratory Gene Therapy Consortium, London, United Kingdom of Great Britain and Northern Ireland.
  • Boyd AC; Washington University School of Medicine in St. Louis, Department of Pediatrics, Division of Allergy & Pulmonary Medicine, St. Louis, Missouri, United States.
  • Donaldson S; Centre for Genomic and Experimental Medicine, Institute of Genetics & Cancer, University of Edinburgh, Edinburgh, United Kingdom of Great Britain and Northern Ireland.
  • Gill DR; UK Respiratory Gene Therapy Consortium, Edinburgh, United Kingdom of Great Britain and Northern Ireland.
  • Griesenbach U; The University of North Carolina at Chapel Hill, Department of Medicine, Division of Pulmonary Diseases and Critical Care Medicine, Chapel Hill, North Carolina, United States.
  • Hyde SC; University of Oxford, Radcliffe Department of Medicine, Oxford, United Kingdom of Great Britain and Northern Ireland.
  • Jain R; UK Respiratory Gene Therapy Consortium, Oxford, United Kingdom of Great Britain and Northern Ireland.
  • McLachlan G; National Heart and Lung Institute, Imperial College London, London, United Kingdom of Great Britain and Northern Ireland.
  • Mall MA; UK Respiratory Gene Therapy Consortium, London, United Kingdom of Great Britain and Northern Ireland.
  • Alton EW; University of Oxford, Radcliffe Department of Medicine, Oxford, United Kingdom of Great Britain and Northern Ireland.
Am J Respir Crit Care Med ; 2024 Sep 05.
Article en En | MEDLINE | ID: mdl-39236265
ABSTRACT
Cystic fibrosis is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. While cystic fibrosis is a multi-organ disease, the leading causes of morbidity and mortality are related to progressive lung disease. Current understanding of the effects of the broad spectrum of CFTR mutations on CFTR function has allowed for the development of CFTR modulator therapies. Despite the remarkable impact that these therapies have had, there remains a significant proportion of people with cystic fibrosis (estimated at 10-15% of the global cystic fibrosis population) who are genetically ineligible for, or intolerant to, current CFTR-targeting therapies and whose therapeutic needs remain unmet. Inhaled genetic therapies offer the prospect of addressing the unmet pulmonary treatment need in people with cystic fibrosis, with several approaches, including gene addition therapy (the focus of this review), RNA-based therapies, antisense oligonucleotides and gene editing, being explored. Various non-viral and viral vectors have been investigated for cystic fibrosis gene addition therapy for mutation-agnostic restoration of CFTR function in the lungs. Lentiviral vectors offer the prospect of highly efficient and long-lasting gene expression, and the potential to be safely and, in contrast to other commonly used viral vectors, effectively re-dosed. A third-generation lentiviral vector pseudotyped with Sendai virus F and HN envelope proteins (rSIV.F/HN) has been developed for the treatment of cystic fibrosis. Promising preclinical results support the progression of this vector carrying a full-length CFTR transgene (BI 3720931) into a first-in-human clinical trial expected to begin in 2024. This article is open access and distributed under the terms of the Creative Commons Attribution Non-Commercial No Derivatives License 4.0 (http//creativecommons.org/licenses/by-nc-nd/4.0/).
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Am J Respir Crit Care Med Asunto de la revista: TERAPIA INTENSIVA Año: 2024 Tipo del documento: Article Pais de publicación: Estados Unidos
Texto completo
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Imprimir
XML
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Am J Respir Crit Care Med Asunto de la revista: TERAPIA INTENSIVA Año: 2024 Tipo del documento: Article Pais de publicación: Estados Unidos